JPWO2021050571A5 - - Google Patents

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Publication number
JPWO2021050571A5
JPWO2021050571A5 JP2022514994A JP2022514994A JPWO2021050571A5 JP WO2021050571 A5 JPWO2021050571 A5 JP WO2021050571A5 JP 2022514994 A JP2022514994 A JP 2022514994A JP 2022514994 A JP2022514994 A JP 2022514994A JP WO2021050571 A5 JPWO2021050571 A5 JP WO2021050571A5
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JP
Japan
Prior art keywords
adenosine deaminase
fusion protein
base editor
protein according
modification
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Application number
JP2022514994A
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English (en)
Japanese (ja)
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JP2022546608A5 (https=
JP7717684B2 (ja
JP2022546608A (ja
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Priority claimed from PCT/US2020/018195 external-priority patent/WO2020168135A1/en
Application filed filed Critical
Priority claimed from PCT/US2020/049975 external-priority patent/WO2021050571A1/en
Publication of JP2022546608A publication Critical patent/JP2022546608A/ja
Publication of JP2022546608A5 publication Critical patent/JP2022546608A5/ja
Publication of JPWO2021050571A5 publication Critical patent/JPWO2021050571A5/ja
Priority to JP2025122950A priority Critical patent/JP2025170240A/ja
Application granted granted Critical
Publication of JP7717684B2 publication Critical patent/JP7717684B2/ja
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JP2022514994A 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法 Active JP7717684B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025122950A JP2025170240A (ja) 2019-09-09 2025-07-23 新規核酸塩基エディター及びその使用方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962897777P 2019-09-09 2019-09-09
US62/897,777 2019-09-09
USPCT/US2020/018195 2020-02-13
PCT/US2020/018195 WO2020168135A1 (en) 2019-02-13 2020-02-13 Compositions and methods for treating alpha-1 antitrypsin deficiency
PCT/US2020/049975 WO2021050571A1 (en) 2019-09-09 2020-09-09 Novel nucleobase editors and methods of using same

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025122950A Division JP2025170240A (ja) 2019-09-09 2025-07-23 新規核酸塩基エディター及びその使用方法

Publications (4)

Publication Number Publication Date
JP2022546608A JP2022546608A (ja) 2022-11-04
JP2022546608A5 JP2022546608A5 (https=) 2023-09-19
JPWO2021050571A5 true JPWO2021050571A5 (https=) 2023-09-19
JP7717684B2 JP7717684B2 (ja) 2025-08-04

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ID=74867196

Family Applications (2)

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JP2022514994A Active JP7717684B2 (ja) 2019-09-09 2020-09-09 新規核酸塩基エディター及びその使用方法
JP2025122950A Pending JP2025170240A (ja) 2019-09-09 2025-07-23 新規核酸塩基エディター及びその使用方法

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JP2025122950A Pending JP2025170240A (ja) 2019-09-09 2025-07-23 新規核酸塩基エディター及びその使用方法

Country Status (8)

Country Link
US (1) US20230075877A1 (https=)
EP (1) EP4028026A4 (https=)
JP (2) JP7717684B2 (https=)
KR (1) KR20220076467A (https=)
CN (1) CN114667149A (https=)
AU (1) AU2020344547A1 (https=)
CA (1) CA3153624A1 (https=)
WO (1) WO2021050571A1 (https=)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
EP3850088A4 (en) 2018-09-07 2023-07-19 Beam Therapeutics, Inc. Compositions and methods for improving base editing
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
US20220235347A1 (en) 2019-02-13 2022-07-28 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
KR20210126680A (ko) * 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CA3214494A1 (en) * 2021-03-26 2022-09-29 Beam Therapeutics Inc. Adenosine deaminase variants and uses thereof
EP4337701A4 (en) 2021-05-10 2025-04-09 Mammoth Biosciences, Inc. EFFECTOR PROTEINS AND METHODS OF USE
WO2023275892A1 (en) * 2021-06-29 2023-01-05 Council Of Scientific & Industrial Research Engineered fncas9 and uses thereof
US20240352439A1 (en) * 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
AU2022342169A1 (en) * 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Hbb-modulating compositions and methods
WO2023052366A1 (en) 2021-09-28 2023-04-06 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of beta-hemoglobinopathies
WO2023099591A1 (en) 2021-12-01 2023-06-08 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer
AU2023209457A1 (en) 2022-01-21 2024-08-08 Mammoth Biosciences, Inc. Effector proteins and methods of use
US20250161492A1 (en) 2022-01-25 2025-05-22 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of beta-thalassemia
AU2023248451A1 (en) 2022-04-04 2024-10-17 President And Fellows Of Harvard College Cas9 variants having non-canonical pam specificities and uses thereof
CN120456933A (zh) 2022-04-28 2025-08-08 布罗德研究所股份有限公司 编码碱基编辑器的aav载体及其用途
EP4522647A1 (en) 2022-05-10 2025-03-19 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the cd39 (cag>tag) mutation in patients suffering from beta-thalassemia
WO2023217904A1 (en) 2022-05-10 2023-11-16 Institut National de la Santé et de la Recherche Médicale Syncitin-1 fusion proteins and uses thereof for cargo delivery into target cells
EP4558633A1 (en) 2022-07-22 2025-05-28 Institut National de la Santé et de la Recherche Médicale Base editing approaches for correcting the ivs2-1 (g>a) mutation in patients suffering from beta-thalassemia
CN115820691B (zh) * 2022-07-25 2023-08-22 安徽农业大学 一种基于LbCpf1变体的水稻碱基编辑系统和应用
CA3263821A1 (en) 2022-08-16 2024-02-22 The Broad Institute, Inc. ADVANCED CYTOSIN DESAMINASE AND DNA EDITING METHODS USING IT
CN116790558A (zh) * 2022-08-22 2023-09-22 山东舜丰生物科技有限公司 突变的Cas12j蛋白及其应用
CN119998447A (zh) * 2022-09-23 2025-05-13 基础科学研究院 新型腺嘌呤脱氨酶变体及使用其进行碱基编辑的方法
JP2026506554A (ja) 2023-02-06 2026-02-25 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 多重塩基編集による遺伝子改変造血幹細胞の濃縮
WO2024230760A1 (zh) * 2023-05-09 2024-11-14 北京齐禾生科生物科技有限公司 一种可作用于dna的腺苷脱氨酶及其应用
CN117965505A (zh) * 2023-06-28 2024-05-03 微光基因(苏州)有限公司 工程化的腺苷脱氨酶及碱基编辑器
AU2024299627A1 (en) 2023-07-25 2026-01-22 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025059520A1 (en) * 2023-09-15 2025-03-20 Beam Therapeutics Inc. Compositions and methods for base editing a phenylalanine hydroxylase polynucleotide
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
CN120536418A (zh) * 2024-02-26 2025-08-26 尧唐(上海)生物科技有限公司 一种脱氨酶变体、包含其的碱基编辑器及其应用

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2753391T3 (es) * 2014-10-14 2020-04-08 Halozyme Inc Composiciones de adenosina desaminasa 2 (ADA2), variantes de la misma y métodos de uso de las mismas
KR102547316B1 (ko) * 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11242542B2 (en) * 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
EP3526320A1 (en) * 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
WO2018089664A1 (en) * 2016-11-11 2018-05-17 The Regents Of The University Of California Variant rna-guided polypeptides and methods of use
KR20250107288A (ko) * 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
CN109957569B (zh) * 2017-12-22 2022-10-25 苏州齐禾生科生物科技有限公司 基于cpf1蛋白的碱基编辑系统和方法
CN109295186B (zh) * 2018-09-30 2023-10-03 中山大学 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用

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